Europe Poised to Approve Hydroxyurea for Sickle Cell Disease

Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended that the European Medicines Agency (EMA) grant marketing authorization to hydroxyurea for the prevention of vaso-occlusive complications of sickle cell disease (SCD) in patients older than age 2.

Hydroxyurea is a ribonucleotide reductase inhibitor that acts by interfering with the synthesis of DNA, without interfering with the synthesis of ribonucleic acid or protein. In the U.S., hydroxyurea initially was approved to treat pain crises in adults with SCD in 1998; in December 2017, the U.S. Food and Drug Administration expanded the indication to include pediatric patients age 2 or older.

According to CHMP’s review of trial data, hydroxyurea reduced the vaso-occlusive complications of SCD. The most common adverse events in patients treated with hydroxyurea included neutropenia, reticulocytopenia, macrocytosis, thrombocytopenia, anemia, headache, dizziness, nausea, and constipation.

The EMA is not bound to abide the CHMP recommendations, but often follows them.

Source: EMA press release, April 26, 2019.